Sarepta rises on long-term Phase IIb DMD data
Sarepta Therapeutics Inc. (NASDAQ:SRPT) jumped $7.17 (22%) to $39.28 on Thursday after reporting long-term follow-up data from Phase IIb trials of eteplirsen ( AVI-4658) to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
Eteplirsen-treated patients demonstrated a significant 151 meter difference in the six-minute walk test (6MWT) at three years compared to a matched natural history control group (p<0.01). Sarepta also said eteplirsen-treated patients showed a slower loss of ambulation than natural history patients; 17% of patients receiving eteplirsen lost ambulation over three years vs. 46% of those in the control group. Sarepta used matched patients from natural history registries to define an external control group, as requested by FDA. ...